Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
REGN7999 · 2 trials · 2 indications
IV cohorts 1 to 4 SC cohorts 1 and 2
IV cohort 5 and SC cohort 3
| Arm | Type | Description |
|---|---|---|
| Part A High Dose | EXPERIMENTAL | Randomized 2:2:1 |
| Part A Low Dose | EXPERIMENTAL | Randomized 2:2:1 |
| Part A Placebo | PLACEBO_COMPARATOR | Randomized 2:2:1 |
| Part B High Dose | EXPERIMENTAL | Randomized 2:2:1 |
| Part B Low Dose | EXPERIMENTAL | Randomized 2:2:1 |
| Part B Placebo | PLACEBO_COMPARATOR | Randomized 2:2:1 |
| IV Cohort 1 | EXPERIMENTAL | Single dose REGN7999 or Placebo; randomized 3:1 |
| IV Cohort 2 | EXPERIMENTAL | Single dose REGN7999 or Placebo; randomized 3:1 |
| IV Cohort 3 | EXPERIMENTAL | Single dose REGN7999 or Placebo; randomized 3:1 |
| IV Cohort 4 | EXPERIMENTAL | Single dose REGN7999 or Placebo; randomized 3:1 |
| IV Cohort 5 | EXPERIMENTAL | Single dose REGN7999 or Placebo; randomized 3:1 |
| SC Cohort 1 | EXPERIMENTAL | Single dose REGN7999 or Placebo; randomized 3:1 |
| SC Cohort 2 | EXPERIMENTAL | Single dose REGN7999 or Placebo; randomized 3:1 |
| SC Cohort 3 | EXPERIMENTAL | Single dose REGN7999 or Placebo; randomized 3:1 |
| Name | Type | Description |
|---|---|---|
| REGN7999 | DRUG | Administered subcutaneous (SC) |
| Placebo | DRUG | Administered SC |
Key Inclusion Criteria: 1. Clinical diagnosis of NTDT as described in the protocol 2. IOL, defined as LIC ≥ 5 mg Fe/g DW as measured by R2\* MRI at screening 3. Serum ferritin ≥ 300 ng/mL as described in the protocol Key Exclusion Criteria: 1. Hemoglobin ≤ 8 g/dL at screening 2. Any RBC transfusi...
REGN7999 is an investigational small molecule being developed by Regeneron Pharmaceuticals for the treatment of iron overload in adult patients with Non-Transfusion Dependent Beta-Thalassemia (NTDT). It is also being studied in healthy participants to assess its safety and how it works in the body.
REGN7999 targets TMPRSS6, a protein involved in iron regulation. By targeting TMPRSS6, the drug aims to reduce iron overload in patients with Non-Transfusion Dependent Beta-Thalassemia (NTDT). This mechanism is being evaluated in clinical trials to determine its safety and efficacy.
REGN7999 is developed by Regeneron Pharmaceuticals, Inc., a biopharmaceutical company traded on NASDAQ under the ticker symbol REGN. The company is conducting clinical trials to evaluate the drug's safety, tolerability, and efficacy in healthy participants and patients with Non-Transfusion Dependent Beta-Thalassemia (NTDT).
REGN7999 is in Phase 2 clinical development. A Phase 1 trial in healthy participants has been completed, and a Phase 2 study in patients with Non-Transfusion Dependent Beta-Thalassemia (NTDT) is active but not recruiting. The drug is investigational and has not been approved by regulatory authorities.
REGN7999 has two clinical trials. NCT05481333 is a completed Phase 1 study in healthy participants in Belgium, with 64 enrolled. NCT06421636 is an active Phase 2 study in adults with Non-Transfusion Dependent Beta-Thalassemia (NTDT) across multiple countries, including the US, Greece, India, and Italy, with 19 enrolled.
REGN7999 is not known to have alternative names. It is a unique investigational small molecule developed by Regeneron Pharmaceuticals. The drug is being studied under its code name REGN7999 in clinical trials for iron overload in Non-Transfusion Dependent Beta-Thalassemia (NTDT).